November 3, 2020

New treatment for a rare obesity

Diabetes drugs known as GLP-1 receptor agonists, such as exenatide (Byetta), are a promising and safe treatment for a rare form of obesity.

Hypothalamic obesity (HO), a complication of a brain tumor called craniopharyngioma, is characterized by excessive weight gain and its cardiometabolic consequences. 

Previous animal and clinical studies have found that glucagon-like peptide-1 receptor agonist (GLP1RA), a drug class that increases insulin release, reduces blood glucose levels and energy intake, and long-term treatment results in weight loss. 

In 2016, Ashley Shoemaker, MD, MSCI, received National Institutes of Health support for a multicenter phase 3 clinical trial of GLP1RA in children and young adults with HO coordinated by researchers at Seattle Children’s Research Institute. 

Forty-two participants were randomized to receive weekly injections of either GLP1RA or placebo for 36 weeks. The percentage of body fat increased in all patients given placebo, whereas body fat decreased in 50% of those treated with GLP1RA. The therapy was well tolerated. 

GLP1RA thus is a promising and safe treatment to improve or stabilize HO in children and young adults, the researchers reported recently in the journal Diabetes, Obesity and Metabolism.

The study was supported by NIH grant DK104936.